this stock is heavily shorted and with FDA approval could get a pop if they get FDA approval soon.
-GENZYME: Genzyme General and Pharming form joint venture to
develop treatment for Pompe's disease
Presswire - October 14, 1998 13:54
Jump to first matched term
M2 PRESSWIRE-14 October 1998-GENZYME: Genzyme General and Pharming form joint venture to develop treatment for
Pompe's disease (C)1994-98 M2 COMMUNICATIONS LTD
Genzyme General (Nasdaq:GENZ) and Pharming Group N.V. (EASDAQ:PHAR) today announced the formation of a joint
venture to develop and commercialize worldwide the enzyme human alpha-glucosidase as a treatment for Pompe's disease, a fatal
lysosomal storage disorder. The companies originally announced their intention to form the joint venture in July, when Genzyme
also made a $14 million equity investment in Pharming.
Under the terms of the agreement, Genzyme will fund up to $14 million of development costs for alpha-glucosidase, which is
produced in the milk of transgenic rabbits. The companies will equally fund joint venture development costs in excess of $14
million. Pharming will receive from Genzyme a one-time $7 million milestone payment on receipt of full approval of a BLA for
alpha- glucosidase by the U.S. Food and Drug Administration.
Pompe's disease is caused by a complete or partial deficiency of the enzyme human alpha-glucosidase. The disease results in a
build-up of glycogen in various muscles and organs of the body, leading to fatal muscle degeneration. Genzyme and Pharming
believe that administration of human alpha-glucosidase to patients suffering from Pompe's disease could alleviate or eliminate
symptoms.
Pompe's disease, also known as acid maltase deficiency or glycogen storage disorder type II, affects an estimated 5,000-10,000
people in the Western world. Clinical forms of Pompe's disease vary according to the age of onset and progression of symptoms.
In June, Pharming announced preliminary results from a completed phase I clinical trial with human alpha-glucosidase. The trial
was designed to determine the safety, tolerability, and pharmacokinetics of the compound in healthy volunteers. Based on the
results obtained in the phase I trial and preclinical studies, the joint venture expects to commence phase II/III clinical trials by the
end of 1998.
In September 1996, Pharming received orphan drug designation for human alpha-glucosidase, which would provide market
exclusivity in the U.S. for seven years following FDA approval.
Genzyme General develops and markets therapeutic and surgical products and diagnostic products and services, including
Cerezyme and Ceredase enzyme replacement therapies for the treatment of Gaucher's disease, a rare, genetic lysosomal storage
disorder. A division of the biotechnology company Genzyme Corp., Genzyme General has its own common stock intended to
reflect its value and track its economic performance.
Pharming focuses on the development, production and worldwide commercialization of human therapeutic proteins, produced at
high levels in the milk of transgenic animals that have been created using the company's proprietary technology.
This press release contains forward-looking statements about potential therapeutic uses and effects of human alpha-glucosidase,
the potential use of transgenic technology, the expected timing of phase II/III clinical trials of human alpha-glucosidase, the
potential market exclusivity for human alpha- glucosidase, the potential market size for human alpha-glucosidase, and a potential
milestone payment to be made upon receipt of FDA approval. Actual results may differ materially depending on the actual
therapeutic effect of human alpha-glucosidase, whether such phase II/III trials are initiated, the actual timing and results of clinical
trials, the actual timing and content of regulatory submissions and decisions of the FDA, and the accuracy of the companies'
information about the size of the Pompe's disease population and competitors' products.
|
